FDA Previews Patient-focused Report on Opioid Use Disorder
The US Food and Drug Administration (FDA) offered a sneak peek Thursday into a new report on the impact of opioid use disorder (OUD) from the perspectives of about 155 patients.The report is set to be released later this year, said FDA operations research analyst Sara Eggers during a joint public workshop between the agency and the Duke Margolis Center for Health Policy at the National Press Club in Washington.
Preliminary results highlighted by Eggers offer insights into the impact of OUD on patients’ day-to-day lives to aid industry and FDA staff in the push for patient-focused drug development.
The input can serve FDA and others in identifying specific areas of unmet needs for patients with OUD, support advice on medical product development programs and facilitate greater appropriate use of approved medications and how these are evaluated, according to Eggers.
FDA committed to issuing disease-specific reports summarizing public meetings under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) in 2012. The initial commitment to conduct 20 meetings on 20 disease areas “has really blossomed into a multi-stakeholder effort to continue to engage patients and their loved ones,” Eggers said, highlighting the completed PDUFA-charted course to issue at least 24 “Voice of the Patient” reports.
Expanding these efforts beyond the PDUFA V commitment with additional disease-specific reports has also enabled the development of “more systematic methodologies to get patients’ experiences, needs and priorities captured meaningfully into drug development,” Eggers added.
The OUD impact report— developed based on the input from participants at FDA’s 17 April public meeting on OUD and comments from a public docket—is one example of the expanded initiative. The docket closed in June after nearly two months, with more than 70 submissions.
Preliminary results
The results highlight the impacts of OUD, a sensation called “craving,” treatment goals, as well as benefits and downsides of and the current barriers to medication-assisted treatment (MAT).
OUD impacts that patients conveyed report feelings of “being a prisoner to intense withdrawals, irritability, skin crawls and spasms, and the obsession with getting their next hit.” Impacts described as “devastating” relate to jobs, careers, school, relationships, well-being and safety. Scars, insomnia, urges, stigma and fear are reportedly at least some of the “lasting” impacts.
With last month’s release of FDA draft guidance on clinical endpoints for demonstrating effectiveness of MAT for OUD, the agency committed to assisting drug sponsors in developing a validated measure of patient-reported experiences such as “craving.”
Participants reported this sensation lasting “well beyond” the intense withdrawal phase that starts out as “an occupying force....of fight or flight” and resurfaces over time.
“[I] used to use all night and I would go to the ATM—[and when I go to the ATM now]—I would still feel the physical sickness about that longing and craving,” a patient said, as cited by Eggers.
When asked about treatment goals, participants stressed the need for an individualized, holistic approach. They also had varying perspectives on their desired outcomes as some desire complete abstinence while others focused on better management of their opioid use. There was consensus, however, around the desired improvement of their own well-being and safety.
Participants’ experience with MAT varied as well. Reported views on the role of MAT ranged from recognizing a need to stay on the treatment regimen or hoping to “detox off MAT” after recovery is stabilized, to seeing no role whatsoever for the currently available options.
Benefits to MAT that patients reported include reducing the “euphoric rush,” as well as being able to “arrest the cravings and compulsiveness” and regain control of their life. In contrast, downsides relate to “intolerable or bothersome” side effects and the need to stay on MAT long-term. MAT also reportedly fails to address underlying issues, such as anxiety and pain.
On challenges to MAT, participants cited a lack of access due to long wait times to obtain medication and strict requirements to remain in a program, among other issues. They also cited unpleasant experiences at MAT facilities, stigma, the intensity of withdrawals, addressing comorbid pain or mental health needs and a self-recognized need to come to terms with OUD.